Precision AQ - Investor Relations & External Communications’ Post

As #CGT continues to grow with significant investment and research, the FDA has created listening meetings for patients of rare diseases and their caregivers to allow them to provide feedback on issues related to approved cell & gene therapy products to support evaluations of short-term and long-term risks, improve industry decision making, and considerations for post-market studies. With the CRISPR Therapeutics approval for CASGEVY, there are now a total of 19 products on the market for 27 indications. With over 1500 clinical trials in progress last Feb and continued investment and focus, that number is expected to expand significantly in the next few years. The FDA continues to take proactive measures to improve communication, capture additional data, and Improve access and safety. Watching the industry grow while bringing hope and benefit to diseases without previous solutions will be exciting while providing incredible opportunities for career growth.  https://lnkd.in/eYv7A_xe #collaboration #biotechnology #riskmanagement #patientadvocate #patientoutcomes #postmarketsurveillance #fda

As part of a recent roundtable discussion, Pharma’s Almanac asked industry leaders what the most significant regulatory decisions on the horizon were. The one that doesn’t happen, quipped Janet Johnson (pictured), the Vice President of Global Regulatory Affairs and Labeling at Terumo Blood and Cell Technologies. “The FDA has long predicted it will approve between 10-20 cell and gene therapies (CGTs) a year by 2025,” she said. “But it will have approved no more than seven by the end of 2023, with at least two decisions coming in after their initial deadlines.” The impact? Sluggish momentum in rolling out critical #CurativeTherapies. Read her commentary and learn what other industry leaders say will be essential regulatory decisions in 2024 and beyond. Check it out now: https://bit.ly/3TxXm73 #FDA #CellTherapies #Healthcare

In this ‘Patti’s People’ episode, Patti Peeples, PhD of the The Peeples Collaborative speaks with Walter Toro Jimenez, MD, PhD, Executive Director, Global HEOR/RWE, Novartis Gene Therapies. Deep diving into patient involvement in the #HTA process. Watch at The Evidence Base® #heor #healtheconomics #outcomesresearch #rwe #rwd #realworldevidence #realworlddata #marketaccess #pharma #biopharma #medicalaffairs #healthtechnologyassessment #healthdata #healthequity #healthpolicy #patientengagement

Brazil on the International Map of Advanced Therapy Product Development. Recently, experts in Advanced Therapy Products (ATPs) from ANVISA (National Health Surveillance Agency) authored a chapter in the book "Regulatory Aspects of Gene Therapy and Cell Therapy Products - A Global Perspective," presenting the regulatory aspects of ATPs in Brazil. Due to their complexity, these products necessitate optimized regulatory pathways for their development and lifecycle monitoring. Approval models for these products in Brazil adapt to the specificities and characteristics of the technology and the patient target population. This includes accelerated regulatory reviews, emergency use provisions, and specific monitoring mechanisms. The incorporation of internationally harmonized concepts aligned with major global regulatory agencies positions Brazil on the international trajectory of ATP development, fostering national growth in this field. Access the complete book to delve further into the regulatory policies for ATPs in Brazil and around the world. #advancedtherapies #regulatory

Clinical research

Exciting collaboration with GenSight Biologics! Be sure to join Scott Jeffers Ph.D. (Chief Technology Officer) from GenSight Biologics at ASGCT #ASGCT2024 May 9 at 12pm. Details below: Poster 1031 Increased Reproducibility in a Critical Downstream Processing Step allows for Speed to Commercialization of Gene Therapy Product to Treat Leber’s Hereditary Optic Neuropathy (LHON) #verdot I #gensightbiologics I #ASGCT2024 I #innovation I #bioprocessing I #singleuse I #purification

Understanding Big Pharma's Pricing formulas for New Medicines. The cost of certain cures, particularly those addressing complex cancers or gene therapy, can be staggering. Contrary to the assumption that these prices are solely justified by extensive research or component scarcity, Big Pharma employs a multifaceted process. They scrutinize a drug's societal value, contemplating factors such as reduced hospital visits, discontinued prescriptions, and gained quality years of life. However, the eventual price tag often appears inflated, with more emphasis on rhetoric than on a robust numerical foundation. #PharmaPricingAnalysis #MedicalCosts #DrugPricingStrategies #HealthcareEconomics #SocietalValueInMedicine #ShareEcard #AJBerman

In the last five years, the FDA approved 12 novel cell and gene therapies. Compare that figure to 3,000 CAGT assets in the global pipeline at the end of 2022. Navigating these constricting pipelines will require careful strategic planning and multidisciplinary expertise. Learn how Parexel can maximize your chances of market success:

FDA Keynote Speaker in the Spotlight, CAR-T Industry Leaders Gather for Discussion-Join the 2024 GenScript Biotech Global Forum for a New Journey in Cell and Gene Therapy: NANJING, China, Dec. 24, 2023 /PRNewswire/ -- The innovative development of biopharmaceuticals has ushered in a new era for disease treatment, bringing unprecedented hope to countless patients. However, the transition from basic research to clinical commercialization presents formidable...

Good exchange yesterday evening in Basel with an enthusiastic group of industry executives. The question: How to close the commercial viability gap in cell and gene therapies? Growth in CGT continues to significantly outpace the pharma market and there will be six blockbuster CGTs in 2028. However, to unlock the full potential, we must 1) become better at identifying the #patients who can benefit, 2) define effective market access and reimbursement strategies to ensure #accessibility, and 3) optimize manufacturing - and start doing so earlier in development. Dr. Oliver Eitelwein, Andreas Eggert, Dr. Clemens Freytag, Allegra Hohn-Honari, Lea Schaad